Lymphangioleiomyomatosis Medication

  • Author: Joel Moss, MD, PhD; Chief Editor: Zab Mosenifar, MD   more...
 
Updated: Apr 2, 2012
 
 

Medication Summary

The goals of pharmacotherapy for lymphangioleiomyomatosis (LAM) are to reduce morbidity and to prevent complications. However, new experimental drug therapies, such as the following, target the underlying disease:

  • Rapamycin: The TSC genes are believed to regulate a protein called mammalian target of rapamycin (mTOR), which is known to control cell growth and proliferation.[33] Rapamycin inhibits the activity of mTOR. In clinical trials, the use of rapamycin was associated with a reduction in the size of renal angiomyolipomas, slowing of the decline in lung function, and resolution of lymphangioleiomyomas and chylous effusions[28, 29, 30, 31]
  • Everolimus (RAD001): This agent is a rapamycin analogue.
  • Doxycycline: Doxycycline, a drug with antibiotic, antiangiogenic, and anti-MMP activities, is being tested for its ability to improve pulmonary function in LAM.
  • Letrozole: Letrozole is an aromatase inhibitor with antiestrogenic effects that is being tested in LAM.
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Contributor Information and Disclosures
Author

Joel Moss, MD, PhD  Deputy Chief, Cardiovascular and Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health

Joel Moss, MD, PhD is a member of the following medical societies: Alpha Omega Alpha, American College of Chest Physicians, American Society for Biochemistry and Molecular Biology, American Society for Clinical Investigation, American Thoracic Society, Association of American Physicians, and Phi Beta Kappa

Disclosure: Nothing to disclose.

Coauthor(s)

Arnold S Kristof, MDCM, FRCPC  Associate Professor of Medicine, Department of Medicine, Respiratory and Critical Care Divisions; Associate Member, Department of Microbiology and Immunology; Research Director, Meakins-Christie Laboratories, McGill University Faculty of Medicine; Attending Physician, Respiratory and Critical Care Divisions, McGill University Health Centre, Royal Victoria Hospital, Canada

Arnold S Kristof, MDCM, FRCPC is a member of the following medical societies: American College of Chest Physicians, American Society for Biochemistry and Molecular Biology, American Thoracic Society, and Royal College of Physicians and Surgeons of Canada

Disclosure: Nothing to disclose.

Specialty Editor Board

Ryland P Byrd Jr, MD  Professor, Department of Internal Medicine, Division of Pulmonary Medicine and Critical Care Medicine, Program Director of Pulmonary Diseases and Critical Care Medicine Fellowship, East Tennessee State University, James H Quillen College of Medicine; Medical Director of Respiratory Therapy, James H Quillen Veterans Affairs Medical Center

Ryland P Byrd Jr, MD is a member of the following medical societies: American College of Chest Physicians and American Thoracic Society

Disclosure: Nothing to disclose.

Francisco Talavera, PharmD, PhD  Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Medscape Salary Employment

Harold L Manning, MD  Professor, Departments of Medicine, Anesthesiology and Physiology, Section of Pulmonary and Critical Care Medicine, Dartmouth Medical School

Harold L Manning, MD is a member of the following medical societies: American College of Chest Physicians, American College of Physicians, and American Thoracic Society

Disclosure: Nothing to disclose.

Timothy D Rice, MD  Associate Professor, Departments of Internal Medicine and Pediatrics and Adolescent Medicine, St Louis University School of Medicine

Timothy D Rice, MD is a member of the following medical societies: American Academy of Pediatrics and American College of Physicians

Disclosure: Nothing to disclose.

Chief Editor

Zab Mosenifar, MD  Director, Division of Pulmonary and Critical Care Medicine, Director, Women's Guild Pulmonary Disease Institute, Professor and Executive Vice Chair, Department of Medicine, Cedars Sinai Medical Center, University of California, Los Angeles, David Geffen School of Medicine

Zab Mosenifar, MD is a member of the following medical societies: American College of Chest Physicians, American College of Physicians, American Federation for Medical Research, and American Thoracic Society

Disclosure: Nothing to disclose.

Additional Contributors

John A Kelly, MB, BCh, MD † Assistant Professor of Medicine and Microbiology and Immunology, Dartmouth Medical School; Staff Pulmonologist, White River Junction Veterans Affairs Medical Center.

Acknowledgments

This work was supported in part by the Division of Intramural Research, National Institutes of Health, National Heart, Lung, and Blood Institute (J.M.) as well as National Institutes of Health R01-CA125436, Tuberous Sclerosis Alliance, LAM Foundation, LAM Canada (A.K.).

References

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